Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol)
← catalyst calendarNCT07740512 · readout in 865 d
Sponsored by Polaryx Therapeutics, Inc. (industry) · PLYX — their whole pipeline →.
Phase
Phase 2
Status
Not yet recruiting
Study type
Interventional
Design
Non randomized · None · Treatment
Enrollment
24estimated
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2026-12-01 | estimated | When the study began enrolling |
| Primary completion | 2028-12-31 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | 2029-02-01 | estimated | The whole study's end, after follow-up |
| First posted | 2026-07-31 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-07-31 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Conditions
- Lysosomal Storage Disorders
- Sandhoff Disease
- Krabbe Disease
- CLN2
- CLN3
Intervention
- Drug: PLX-200 — also filed as Gemfibrozil
Primary outcomes
what the primary-completion date above is the date OFTo evaluate the safety and tolerability of PLX-200 in study participants 2 to 15 years old1 with LSDs2 during the treatment period.
measured Until 30 days after the last administration of the study drug.
To evaluate the safety and tolerability of PLX-200 in study participants 2 to 15 years old1 with LSDs2 during the treatment period.
measured 30 days after the last administration of the study drug
To evaluate the change from baseline in clinical laboratory parameters in study participants 2 to 15 years old with LSDs during the treatment period.
measured Week 102
To evaluate the change from baseline in physical examination results in study participants 2 to 15 years old with LSDs during the treatment period.
measured Week 102
To evaluate the change from baseline vital signs in study participants 2 to 15 years old with LSDs during the treatment period.
measured Week 102
To evaluate the change from baseline in 12-lead electrocardiogram in study participants 2 to 15 years old with LSDs during the treatment period.
measured Week 102
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